Modulation of dystrophia myotonica-protein kinase (DMPK) expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsC. Frank Bennett, Susan M. Freier, Robert A. MacLeod, Sanjay K. Pandey, Charles A. Thornton, Thurman Wheeler, Seng H. Cheng, Andrew Leger, Bruce M. Wentworth
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Abstract
Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
Claims24
A method of treating an animal having type 1 myotonic dystrophy comprising administering to the animal a therapeutically effective amount of a compound comprising an oligonucleotide consisting of 10 to 30 linked nucleosides targeted to a DMPK nucleic acid, wherein the oligonucleotide has a nucleobase sequence that is at least 90% complementary to a non-CUG repeat region of SEQ ID NO:
- 1 or SEQ ID NO:
- 2 as measured over the entirety of the oligonucleotide, wherein the compound reduces myotonia or reduces spliceopathy in the animal.
Dependencies are inferred from the claim text.